Biogen shares FDA decisions on ALS drug

pharmafile | March 23, 2023 | News story | Research and Development  

US-based Biogen has announced two decisions the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee made surrounding its ALS drug, tofersen. The FDA will continue its review of tofersen with a Prescription Drug User Fee Act action date of 25 April, 2023.


In superoxide dismutase 1 (SOD1)-ALS, mutations in the SOD1 gene mean the body creates a toxic form of SOD1 protein. Tofersen is an antisense oligonucleotide (ASO), designed to bind SOD1 mRNA and reduce SOD1 protein production.


Tofersen is being studied in the open label extension of the phase 3 VALOR study and the phase 3 ATLAS study. ATLAS is evaluating whether tofersen can delay clinical onset when initiated in presymptomatic individuals with SOD1 genetic mutation and biomarker evidence of disease activity.


The first question posed to the Committee was: “Is the available evidence sufficient to conclude that a reduction in plasma neurofilament light chain (NfL) concentration in tofersen-treated patients is reasonably likely to predict clinical benefit of tofersen for treatment of patients with SOD1-ALS?” The Committee voted unanimously yes (nine yes, zero no) for consideration of a potential accelerated approval.


The second, however, revealed a more divisive opinion. “Does the clinical data from the placebo-controlled study and available long-term extension study results, with additional supporting results from the effects on relevant biomarkers (ie, changes in plasma NfL concentration and/or reductions in SOD1), provide substantial evidence of the effectiveness of tofersen in the treatment of patients with SOD1-ALS?” split the Committee into three yes, five no and one abstain for consideration of a potential traditional approval.


Priya Singhal, MD, MPH, executive VP, head of development and interim head of research and global safety and regulatory sciences at Biogen, stated: “After hearing the moving experiences of the ALS community and reviewing the totality of data, the Committee voted that reductions of neurofilament are reasonably likely to predict clinical benefit of tofersen. If approved, tofersen would potentially represent a major advance for people living with SOD1-ALS. We thank the FDA for convening this important discussion. Most importantly, we are grateful to all the people with SOD1-ALS who participated in our tofersen studies, and their caregivers, families, study investigators and the entire community, without whom this scientific progress could not have been made.”


James Spargo

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